PL2191001T3 - Raav vector compositions having tyrosine-modified capsid proteins and methods for use - Google Patents

Raav vector compositions having tyrosine-modified capsid proteins and methods for use

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Publication number
PL2191001T3
PL2191001T3 PL08733161T PL08733161T PL2191001T3 PL 2191001 T3 PL2191001 T3 PL 2191001T3 PL 08733161 T PL08733161 T PL 08733161T PL 08733161 T PL08733161 T PL 08733161T PL 2191001 T3 PL2191001 T3 PL 2191001T3
Authority
PL
Poland
Prior art keywords
tyrosine
methods
capsid proteins
raav vector
modified capsid
Prior art date
Application number
PL08733161T
Other languages
Polish (pl)
Other versions
PL2191001T4 (en
Inventor
Li Zhong
Sergei Zolotukhin
Lakshmanan Govindasamy
Mavis Agbandje-Mckenna
Arun Srivastava
Original Assignee
University Of Florida Research Foundation, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University Of Florida Research Foundation, Inc. filed Critical University Of Florida Research Foundation, Inc.
Publication of PL2191001T3 publication Critical patent/PL2191001T3/en
Publication of PL2191001T4 publication Critical patent/PL2191001T4/en

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/515—Animal cells
    • A61K2039/5158—Antigen-pulsed cells, e.g. T-cells
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

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  • Manufacturing & Machinery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Oncology (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
PL08733161T 2007-04-09 2008-04-08 Raav vector compositions having tyrosine-modified capsid proteins and methods for use PL2191001T4 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US91079807P 2007-04-09 2007-04-09

Publications (2)

Publication Number Publication Date
PL2191001T3 true PL2191001T3 (en) 2017-01-31
PL2191001T4 PL2191001T4 (en) 2017-01-31

Family

ID=39684174

Family Applications (1)

Application Number Title Priority Date Filing Date
PL08733161T PL2191001T4 (en) 2007-04-09 2008-04-08 Raav vector compositions having tyrosine-modified capsid proteins and methods for use

Country Status (13)

Country Link
US (11) US8445267B2 (en)
EP (3) EP2623605B1 (en)
CA (1) CA2720097C (en)
CY (1) CY1118228T1 (en)
DK (2) DK2191001T3 (en)
ES (3) ES2714007T3 (en)
HR (1) HRP20161150T1 (en)
HU (1) HUE030719T2 (en)
LT (2) LT2191001T (en)
PL (1) PL2191001T4 (en)
PT (1) PT2191001T (en)
SI (1) SI2191001T1 (en)
WO (1) WO2008124724A1 (en)

Families Citing this family (116)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2191001T3 (en) * 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8840881B2 (en) * 2008-08-28 2014-09-23 Aduro Gvax Inc. Methods and compositions for treating prostate cancer or inducing a humoral immune response against prostate cancer
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
GB2492719A (en) 2010-04-05 2013-01-09 Eos Neuroscience Inc Methods and compositions for decreasing chronic pain
JP2013533847A (en) 2010-04-23 2013-08-29 ユニバーシティ オブ マサチューセッツ AAV-based treatment of cholesterol-related disorders
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
CA3049237C (en) 2010-04-23 2024-06-11 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
CN103189507A (en) 2010-10-27 2013-07-03 学校法人自治医科大学 Adeno-associated virus virions for transferring genes into neural cells
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
US20140162319A2 (en) * 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
US9677088B2 (en) * 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
KR102298579B1 (en) * 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
AU2014324717B2 (en) 2013-09-26 2020-10-08 University Of Florida Research Foundation, Inc. Synthetic combinatorial AAV capsid library for targeted gene therapy
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
WO2015126972A1 (en) * 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
WO2015134643A1 (en) * 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
GB2526339A (en) * 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
JP6842410B2 (en) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ AAV vector identified by a novel high efficiency library
RU2020140209A (en) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс RECOMBINANT AAV OPTIONS AND THEIR APPLICATIONS
MX2017005834A (en) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease.
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
RU2749882C2 (en) 2014-11-14 2021-06-18 Вояджер Терапьютикс, Инк. Modulating polynucleotides
KR102599909B1 (en) 2014-11-14 2023-11-09 보이저 테라퓨틱스, 인크. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP3221456B1 (en) 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
WO2016134337A1 (en) * 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Site-specific integrating recombinant aav vectors for gene therapy and improved production methods
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions and uses thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
ES2865487T3 (en) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Methods and compositions for viral vectors that evade antibodies
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
CA3002982A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
GB201519086D0 (en) 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
CA3006569A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
JP6996728B2 (en) * 2016-01-07 2022-01-17 学校法人自治医科大学 Adeno-associated virus vector for glucose transporter 1 expression
MX2018008651A (en) 2016-01-13 2019-03-14 Merial Inc Recombinant aav vectors expressing osteoprotective genes, including has2 and lubricin, useful in the treatment of osteoarthritis and related joint conditions in mammals.
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
JP7364306B2 (en) 2016-03-31 2023-10-18 スパーク セラピューティクス インコーポレイテッド Column-based highly scalable rAAV manufacturing process
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN116333057A (en) 2016-05-13 2023-06-27 4D分子治疗有限公司 Adeno-associated virus variant capsids and methods of use thereof
WO2017201121A1 (en) * 2016-05-17 2017-11-23 University Of Florida Research Foundation, Inc. Recombinant aav for gene therapy in lungs
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc Modulatory polynucleotides
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
US10961556B2 (en) 2016-08-15 2021-03-30 Danmarks Tekniske Universitet Method for reducing ammonium and lactate production in CHO cells
AU2017321488B2 (en) 2016-08-30 2022-10-20 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10650621B1 (en) 2016-09-13 2020-05-12 Iocurrents, Inc. Interfacing with a vehicular controller area network
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
KR20190075964A (en) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 AAV capsid design
EP3569708A4 (en) 2017-01-13 2020-09-09 Jichi Medical University Aav vector for disrupting clotting-related factor gene on liver genome
US11028131B2 (en) 2017-01-30 2021-06-08 Nippon Medical School Foundation Mutant of adeno-associated virus (AAV) capsid protein
CA3054131C (en) 2017-02-21 2021-05-25 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
MX2019013172A (en) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease.
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CN111132626B (en) 2017-07-17 2024-01-30 沃雅戈治疗公司 Track array guidance system
KR20220070075A (en) 2017-09-20 2022-05-27 4디 몰레큘러 테라퓨틱스 아이엔씨. Adeno-associated virus variant capsids and methods of use thereof
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
TWI804518B (en) 2017-10-16 2023-06-11 美商航海家醫療公司 Treatment of amyotrophic lateral sclerosis (als)
CN111770999A (en) 2017-11-27 2020-10-13 4D分子治疗有限公司 Adeno-associated virus variant capsids and use for inhibiting angiogenesis
US10806802B2 (en) 2018-01-18 2020-10-20 University Of Guelph Adeno-associated virus particle with mutated capsid and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019161365A1 (en) 2018-02-19 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
CN112272672A (en) 2018-04-03 2021-01-26 斯特里迪比奥公司 Antibody-evasive viral vectors
WO2019210249A1 (en) * 2018-04-26 2019-10-31 Oregon Health & Science University Methods of aav vector production by modulating deubiquitinating enzyme activity
US20210363192A1 (en) * 2018-04-27 2021-11-25 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
US20210230229A1 (en) * 2018-05-15 2021-07-29 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
JPWO2020026968A1 (en) 2018-07-30 2021-08-12 株式会社遺伝子治療研究所 Method of enhancing gene expression by AAV vector
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201821082D0 (en) 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
GB201821089D0 (en) 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
JP2022521025A (en) 2019-02-25 2022-04-04 ノバルティス アーゲー Compositions and Methods for Treating Bietti Crystallin Retinopathy
JP2022520875A (en) 2019-02-25 2022-04-01 ノバルティス アーゲー Compositions and Methods for Treating Bietti Crystallin Retinopathy
AR118465A1 (en) 2019-03-21 2021-10-06 Stridebio Inc RECOMBINANT ADENO-ASSOCIATED VIRUS VECTORS
GB201906052D0 (en) 2019-04-30 2019-06-12 Int Centre For Genetic Engineering And Biotechnology Proteins with cardioprotective activity
CA3145309A1 (en) 2019-07-10 2021-01-14 Locanabio, Inc. Rna-targeting knockdown and replacement compositions and methods for use
EP4006160A4 (en) 2019-07-12 2023-06-28 Gene Therapy Research Institution Co., Ltd. Adeno-associated virus virion for gene transfer to human liver
US20220347298A1 (en) 2019-10-04 2022-11-03 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for treatment of niemann-pick disease type c
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
WO2023172491A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
TW202142552A (en) 2020-01-29 2021-11-16 美商健臻公司 Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
EP4103214A2 (en) 2020-02-13 2022-12-21 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
CA3164714A1 (en) 2020-02-14 2021-08-19 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
US20230235353A1 (en) 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
EP4127189A1 (en) 2020-03-31 2023-02-08 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
MX2023004377A (en) 2020-10-16 2023-06-28 Gyroscope Therapeutics Ltd Nucleic acid encoding an anti-vegf entity and a negative complement regulator and uses thereof for the treatment of age-related macular degeneration.
JP2023551873A (en) 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド RNA targeting compositions and methods for treating CAG repeat disease
EP4255471A1 (en) 2020-12-01 2023-10-11 Locanabio, Inc. Rna-targeting compositions and methods for treating myotonic dystrophy type 1
WO2022221278A1 (en) 2021-04-12 2022-10-20 Locanabio, Inc. Compositions and methods comprising hybrid promoters
CN113121655B (en) * 2021-04-19 2021-11-19 上海信致医药科技有限公司 Ocular and muscle specific targeting type adeno-associated virus vector and application thereof
EP4328313A1 (en) 2021-04-21 2024-02-28 Jichi Medical University Adeno-associated virus virion for treating ornithine transcarbamylase deficiency
JP2024523707A (en) 2021-07-08 2024-06-28 テナヤ セラピューティクス， インコーポレイテッド Optimized Expression Cassettes for Gene Therapy
EP4384196A1 (en) 2021-08-11 2024-06-19 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
WO2023101281A1 (en) * 2021-11-30 2023-06-08 연세대학교 산학협력단 Mutant of adeno-associated virus capsid protein
WO2023106261A1 (en) 2021-12-06 2023-06-15 学校法人自治医科大学 Recombinant adeno-associated virus vector for treatment of iron-accumulating neurodegenerative diseases
WO2023154807A2 (en) 2022-02-09 2023-08-17 Locanabio, Inc. Compositions and methods for modulating pre-mrna splicing
US20230279397A1 (en) 2022-03-04 2023-09-07 Locanabio, Inc. Compositions and methods comprising engineered short nuclear rna (snrna)
WO2023205637A1 (en) 2022-04-18 2023-10-26 Locanabio, Inc. Rna-targeting compositions and methods for treating c9/orf72 diseases
WO2023250447A2 (en) * 2022-06-22 2023-12-28 Synvivia, Inc. Methods of selection and use of engineered cyclic peptides for enhancing viral production
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
WO2024086650A1 (en) 2022-10-18 2024-04-25 Locanabio, Inc. Compositions and methods comprising programmable snrnas for rna editing
WO2024119102A1 (en) 2022-12-01 2024-06-06 Locanabio, Inc. Adeno-associated viral vectors for proper packaging of repetitive elements

Family Cites Families (96)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US102A (en) 1861-08-13 Whole
US103A (en) 1836-12-10 Process
US106A (en) 1915-08-24 Specipication
US105A (en) 1836-12-15 knight
US104A (en) 1836-12-10 Revolving hokse-rake
EP0003048B2 (en) 1978-01-05 1983-08-24 Bayer Ag Molding compositions consisting essentially of unsaturated polyesters, method of preparation and application
US4216209A (en) 1979-03-19 1980-08-05 American Home Products Corporation Tripeptide angiotensin converting enzyme inhibitors
US4554101A (en) 1981-01-09 1985-11-19 New York Blood Center, Inc. Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity
US4883750A (en) 1984-12-13 1989-11-28 Applied Biosystems, Inc. Detection of specific sequences in nucleic acids
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4800159A (en) 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US5116742A (en) 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
US5037746A (en) 1986-12-03 1991-08-06 University Patents, Inc. RNA ribozyme polymerases, and methods
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
AU622104B2 (en) 1987-03-11 1992-04-02 Sangtec Molecular Diagnostics Ab Method of assaying of nucleic acids, a reagent combination and kit therefore
IL86724A (en) 1987-06-19 1995-01-24 Siska Diagnostics Inc Method and kits for the amplification and detection of nucleic acid sequences
AU622426B2 (en) 1987-12-11 1992-04-09 Abbott Laboratories Assay using template-dependent nucleic acid probe reorganization
AU624601B2 (en) 1988-01-21 1992-06-18 Genentech Inc. Amplification and detection of nucleic acid sequences
CA1340807C (en) 1988-02-24 1999-11-02 Lawrence T. Malek Nucleic acid amplification process
US4932207A (en) 1988-12-28 1990-06-12 Sundstrand Corporation Segmented seal plate for a turbine engine
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
US5219727A (en) 1989-08-21 1993-06-15 Hoffmann-Laroche Inc. Quantitation of nucleic acids using the polymerase chain reaction
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
WO1991005041A1 (en) 1989-09-26 1991-04-18 Townes Tim M Erythroid-specific gene expression system
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
JPH03251543A (en) 1990-02-26 1991-11-11 Unitika Ltd Angiotensin conversion enzyme-inhibiting agent
EP0445606B1 (en) 1990-02-27 1997-01-22 The Agency of Industrial Science and Technology Novel oligopeptides, pharmaceutical composition and food containing the same, and use of oligopeptides
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
DE69108553T2 (en) 1990-06-22 1995-08-03 Fisons Plc INHIBITORS FOR ANGIOTENSIN CONVERTING ENZYMS.
JP3218637B2 (en) 1990-07-26 2001-10-15 大正製薬株式会社 Stable aqueous liposome suspension
JP2958076B2 (en) 1990-08-27 1999-10-06 株式会社ビタミン研究所 Multilamellar liposome for gene transfer and gene-captured multilamellar liposome preparation and method for producing the same
US5145684A (en) 1991-01-25 1992-09-08 Sterling Drug Inc. Surface modified drug nanoparticles
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5455166A (en) 1991-01-31 1995-10-03 Becton, Dickinson And Company Strand displacement amplification
DE4216134A1 (en) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog SYNTHETIC CATALYTIC OLIGONUCLEOTIDE STRUCTURES
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
EP0620849B1 (en) 1992-01-07 2003-06-25 Elan Pharmaceuticals, Inc. Transgenic animal models for alzheimer's disease
US5552397A (en) 1992-05-18 1996-09-03 E. R. Squibb & Sons, Inc. Substituted azepinone dual inhibitors of angiotensin converting enzyme and neutral exdopeptidase
US5646020A (en) 1992-05-14 1997-07-08 Ribozyme Pharmaceuticals, Inc. Hammerhead ribozymes for preferred targets
ES2105262T3 (en) 1992-05-18 1997-10-16 Minnesota Mining & Mfg PHARMACY SUPPLY DEVICE THROUGH THE MUCOSAS.
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
US5297721A (en) 1992-11-19 1994-03-29 Fry's Metals, Inc. No-clean soldering flux and method using the same
US5639655A (en) 1993-01-19 1997-06-17 Ribozyme Pharmaceuticals, Inc. PML-RARA targeted ribozymes
US5279721A (en) 1993-04-22 1994-01-18 Peter Schmid Apparatus and method for an automated electrophoresis system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5639940A (en) 1994-03-03 1997-06-17 Pharmaceutical Proteins Ltd. Production of fibrinogen in transgenic animals
US5648211A (en) 1994-04-18 1997-07-15 Becton, Dickinson And Company Strand displacement amplification using thermophilic enzymes
US5789655A (en) 1994-05-13 1998-08-04 The Regents Of The University Of California Transgenic animals expressing artificial epitope-tagged proteins
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
US5580579A (en) 1995-02-15 1996-12-03 Nano Systems L.L.C. Site-specific adhesion within the GI tract using nanoparticles stabilized by high molecular weight, linear poly (ethylene oxide) polymers
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5646031A (en) 1995-05-16 1997-07-08 Northern Illinois University SArMV and sCYMVI hairpin ribozymes
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
US5863736A (en) 1997-05-23 1999-01-26 Becton, Dickinson And Company Method, apparatus and computer program products for determining quantities of nucleic acid sequences in samples
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US7052692B1 (en) 1997-09-02 2006-05-30 Advanced Research & Technology Institute Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression
JP2004534543A (en) 2001-07-13 2004-11-18 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン Pseudotyped adeno-associated virus and uses thereof
NZ600121A (en) * 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2664505T3 (en) * 2001-12-17 2018-04-19 The Trustees Of The University Of Pennsylvania Sequences of serotype 9 of adeno-associated virus (AAV), vectors containing them, and uses thereof
WO2003087324A2 (en) 2002-04-09 2003-10-23 Children's Hospital, Inc. Antibody gene transfer and recombinant aav
AU2003221733A1 (en) 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
WO2004027019A2 (en) 2002-05-01 2004-04-01 University Of Florida Research Foundation, Inc. Improved raav expression systems for genetic modification of specific capsid proteins
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
SI2292780T1 (en) * 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525143T3 (en) 2005-04-07 2014-12-18 The Trustees Of The University Of Pennsylvania AAVrh.64 modified capsules, compositions containing them and uses thereof
US20090215870A1 (en) 2005-04-29 2009-08-27 Terwilliger Ernest F Compositions and methods for targeting of viral vectors
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US8312249B1 (en) 2008-10-10 2012-11-13 Apple Inc. Dynamic trampoline and structured code generation in a signed code environment
FR2957821B1 (en) 2010-03-24 2014-08-29 Inst Francais Du Petrole NEW AREA OF CATALYST REGENERATION DIVIDED IN SECTORS FOR REGENERATIVE CATALYTIC UNITS
CA3049237C (en) 2010-04-23 2024-06-11 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
CN103189507A (en) 2010-10-27 2013-07-03 学校法人自治医科大学 Adeno-associated virus virions for transferring genes into neural cells
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
PL2839014T3 (en) 2012-04-18 2021-08-23 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
US20230136849A1 (en) * 2012-05-15 2023-05-04 University Of Florida Research Foundation, Incorporated Capsid-modified raav vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
CN103060331B (en) 2012-12-05 2014-10-08 南京医科大学第一附属医院 Preparation method and application of capsid-protein-mutant double-stranded recombinant adeno-associated virus containing mediated-membrane-stabile CD40L gene
WO2015134643A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
CA3234028A1 (en) * 2014-03-11 2015-09-17 Wayne State University A modified mglur6 promoter and methods of use
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US20180135074A1 (en) 2015-02-19 2018-05-17 University Of Florida Research Foundation, Incorporated Recombinant aav vectors for gene therapy of human hematopoietic disorders
US20190000943A1 (en) 2015-08-13 2019-01-03 Aavet Therapeutics, Llc Aav6 vectors for immunotherapy

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